MyPART: My Pediatric and Adult Rare Tumor Network - Cures

Division Of Basic Sciences - Nci

Investigators

Linked publications & trials

Abstract

The natural history of rare solid tumors study (19-C-0016) (NHRST) at CCR (PI Dr. Mary Frances Wedekind Malone) has been the backbone of the MyPART program. The goals of the study are to 1) comprehensively and longitudinally evaluate rare tumors in children and young adults through 2) standardized evaluations, medical record extraction, patient reported outcomes, comprehensive clinical and research molecular profiling, with 3) return of results and recommendations including identification of suitable clinical trials after molecular tumor boards, with help of a genetic counselor. An annotated biospecimen repository is established and data are deposited in NIH approved databases led by Dr. Reilly's group. An important aspect of this study is that patients can enroll remotely or at the NIH Clinical Center. This allowed enrolling patients during the Covid 19 pandemic and enabled international participation. Data collection includes standardized web-based forms for family and medical history, patient reported outcomes, tumor pathology, tumor treatment history, tumor imaging, and biospecimen collection. This study (19-C-0016) opened for enrollment in January 2019 and has enrolled 747 participants as of 7/15/2025 with >68 histologies from 46 US states and 27 countries outside the US. For the most frequent histologies enrolled dedicated clinical disease champions have been identified: Chordoma (N=136), Dr. Wedekind Malon, gastrointestinal stromal tumor (non-kit or PDGFRA mutated) (N=113), Dr. Glod, neuroendocrine tumors (N=99), Dr. Del Rivero, adrenocortical carcinoma (N=73), Dr. Del Rivero, medullary thyroid carcinoma (N=36), Dr. Glod. Given pediatric and adult oncology expertise at CCR, patients of any age with these tumors can enroll, allowing the evaluation of biologic differences of tumors in children and adults. Making this protocol available to PIs in CCR, Dr. Alewine (former, CCR medical oncology) has been able to enroll participants with very rare pancreatic carcinoma without the need to develop a new study. The NHRST study is open to enrollment at Oregon Health Science University, Portland OR (PI, Dr. Shivaani Kummar), 4 patients enrolled, and at Texas Children's Hospital, Houston, TX (PI, Dr. Rajkumar Venkatramani), 29 patients enrolled. In addition to the biospecimens used for clinical care, biospecimens have been obtained on many of the patients for molecular research studies (deep whole genome sequencing, total RNA sequencing, MiRNA sequencing, DNA methylation array) from tumor specimens and flow cytometry and cytokine analyses from peripheral blood samples. By this end of this fiscal year, molecular assays will be completed for most of the participants. The results of these assays are in the process of quality control and analyses. Resulting molecular and clinical data are being submitted to public databases in a timely fashion. During the conduct of the study, we have learned that focus on a limited number of rare tumors is needed to enroll cohorts, which may be sufficiently large and well characterized to serve as potential external controls for interventional clinical trials. We have thus collaborated with the NCI Childhood Cancer Data Initiative (CCDI), the Children's Oncology Group (COG), non-COG investigators and advocacy towards the development of an NCI Childhood Cancer Data Initiative (CCDI)-led Pediatric, Adolescent, and Young Adult Rare Cancer Registry. This observational longitudinal will allow studying multiple rare tumors simultaneously. Eligible patients will be identified through COG-APEC14B1 and community engagement. The foundational infrastructure of this study will be provided to disease champions across the country. The protocol has been developed with the goal to open enrollment in the fall of 2025. With the CCDI led study beginning, the POB NHRST study will focus on studying fewer tumor types and allow enrollment of patients not eligible for the CCDI coordinated study due to age (>25 years old). Finally, the POB NHRST will continue to host NIH rare tumor clinics for tumors of interest including tumors selected by the CCDI-coordinated effort.